Title: CARDS: CRISPR Arrayed Repeat Detection System / Part I. UMMS15-58; Patent Pending.
This invention discloses a platform to detect DNA repeat expansions called CRISPR Arrayed Repeat Detection System (CARDS). Utilizing this platform primary cell cultures and/or blood cell smears can be tested under conventional clinical diagnostic laboratory conditions to diagnose genetically-based diseases having DNA repeat expansions, including but not limited to ALS. Further disclosed are dCas9 constructs having fluorescent proteins bound to any or all stem loop sequences, wherein detection of a plurality of dCas9 constructs having different colored fluorescent proteins can simultaneously detect at least six (6) different gene target loci.
Title: ANTI-SOD1 ANTIBODIES AND USES THEREOF. UMMS10-69; Patent 9,109,037
This invention discloses a panel of newly discovered human monoclonal antibodies against human SOD1 protein. There are two types of SOD1 antibodies available: 1) one that binds to all forms of SOD1 and 2) one that binds to the misfolded form of SOD1. Mutated SOD1 is associated with 20% of inheritable forms of Amyotrophic Lateral Sclerosis (ALS) and blocking its function is protective. These monoclonal antibodies present a unique therapeutic opportunity to treat ALS with an immunologically targeted approach.
Title: CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF 1. UMMS10-38. Patent 9,102,949
New effective and safe gene therapy approach that allows gene delivery across the blood brain barrier (BBB) upon intravascular administration. Designed to minimize off-target effects by incorporation of non-CNS-tissue specific miRNA binding site into the transgene expression cassette.
Title: CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF 2. UMMS10-36; Patent 9,102,949
AAV10 vector-based gene therapy for treating ALS with enhanced tropism for neuronal cells. Administration of rAAV encoding inhibitory RNA for superoxide dismutase 1 (SOD1) distributes widely throughout CNS with low toxicity. Long-term inhibition of mutant SOD1 improves lifespan in the animal models.
Innovation Topics: Diagnostics, Neurology, Amyolateral Sclerosis (ALS), CRISPR, Gene editing, Biologics, Neuronal SOD1, Human monoclonal, Antibody, Gene Therapy, Neurodegerative disorder, Canavan Disease, AAV, Gene therapy improvement